Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

Registered drugNo placebo

Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

Registered drugNo placebo

Germany

Participants:0–17 years
Substances:Busulfan

Contact Research Centre

Sickle cell anaemia+1

Recruiting

Study on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease

Registered drugNo placebo

Study on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease

Registered drugNo placebo

Germany

Participants:0–17 years · 18–64 years
Substances:Busulfan

Contact Research Centre

Immunodeficiency

Recruiting

Study on the Use of OTL-103 Gene Therapy and Drug Combination for Patients with Wiskott-Aldrich Syndrome

Registered drugNo placebo

Study on the Use of OTL-103 Gene Therapy and Drug Combination for Patients with Wiskott-Aldrich Syndrome

Registered drugNo placebo

Italy

Participants:0–17 years · 18–64 years
Substances:Busulfan

Contact Research Centre

Sickle cell anaemia

Not yet recruiting

Study on the Effectiveness and Safety of Exa-cel for Adolescents and Adults with Severe Sickle Cell Disease

Registered drugNo placebo

Study on the Effectiveness and Safety of Exa-cel for Adolescents and Adults with Severe Sickle Cell Disease

Registered drugNo placebo

France

Participants:0–17 years · 18–64 years
Substances:Busulfan

Contact Research Centre

Mucopolysaccharidosis I

Not recruiting

Study on Gene Therapy with Autologous Stem Cells and Drug Combination for Children with Mucopolysaccharidosis Type I Hurler Variant

Registered drugNo placebo

Study on Gene Therapy with Autologous Stem Cells and Drug Combination for Children with Mucopolysaccharidosis Type I Hurler Variant

Registered drugNo placebo

Italy

Participants:0–17 years
Substances:Busulfan

Contact Research Centre

Pyruvate kinase deficiency anaemia

Not recruiting

Study on Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children

Registered drugNo placebo

Study on Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children

Registered drugNo placebo

Spain

Participants:0–17 years · 18–64 years
Substances:Busulfan

Contact Research Centre

Sickle cell anaemia

Not recruiting

Study of Exagamglogene Autotemcel (CTX001) for Treatment of Severe Sickle Cell Disease Using Modified Stem Cells

Registered drugNo placebo

Study of Exagamglogene Autotemcel (CTX001) for Treatment of Severe Sickle Cell Disease Using Modified Stem Cells

Registered drugNo placebo

Belgium

Participants:0–17 years · 18–64 years
Substances:Busulfan

Contact Research Centre

Thalassaemia beta

Not recruiting

Study on the Safety and Effectiveness of CRISPR-Cas9 Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel

Registered drugNo placebo

Study on the Safety and Effectiveness of CRISPR-Cas9 Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel

Registered drugNo placebo

Germany

Participants:0–17 years · 18–64 years
Substances:Busulfan

Contact Research Centre

Renal cancer metastatic

Not recruiting

Study on the Safety and Effects of Temferon for Patients with Metastatic Renal Cell Carcinoma Using a Drug Combination

Registered drugNo placebo

Study on the Safety and Effects of Temferon for Patients with Metastatic Renal Cell Carcinoma Using a Drug Combination

Registered drugNo placebo

Italy

Participants:18–64 years · 65+ years
Substances:Busulfan

Contact Research Centre

Find therapies in clinical trials near you

13 trials match your criteria

Thalassaemia beta+1

A Study of Gene Therapy Using Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Who Need Regular Blood Transfusions

Osteopetrosis

Study of gene-modified stem cells (FT024) in children with TCIRG1-related osteopetrosis using treosulfan, thiotepa, and plerixafor for treatment

Sickle cell anaemia

Study on the Safety and Effectiveness of CTX001 for Children with Severe Sickle Cell Disease Using Exagamglogene Autotemcel, Busulfan, and Plerixafor

Thalassaemia

Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

Sickle cell anaemia+1

Study on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease

Immunodeficiency

Study on the Use of OTL-103 Gene Therapy and Drug Combination for Patients with Wiskott-Aldrich Syndrome

Sickle cell anaemia

Study on the Effectiveness and Safety of Exa-cel for Adolescents and Adults with Severe Sickle Cell Disease

Mucopolysaccharidosis I

Study on Gene Therapy with Autologous Stem Cells and Drug Combination for Children with Mucopolysaccharidosis Type I Hurler Variant

Pyruvate kinase deficiency anaemia

Study on Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children

Sickle cell anaemia

Study of Exagamglogene Autotemcel (CTX001) for Treatment of Severe Sickle Cell Disease Using Modified Stem Cells

Thalassaemia beta

Study on the Safety and Effectiveness of CRISPR-Cas9 Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel

Renal cancer metastatic

Study on the Safety and Effects of Temferon for Patients with Metastatic Renal Cell Carcinoma Using a Drug Combination

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